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使用AAV44.9载体的全身基因疗法拯救了一个新生儿致命的小鼠模型的propionic acidemia

Randy J Chandler1, Giovanni Di Pasquale2, Eun-Young Choi1

  • 1National Human Genome Research Institute, Bethesda, MD 20892, USA.

Molecular therapy. Methods & clinical development
|September 25, 2023
PubMed
概括

使用AAV44.9载体的基因疗法对 propionic acidemia (PA) 的治疗有希望. 一次新生儿剂量改善了新PA小鼠模型中的生存率和减少了有毒代谢物,突出了代谢障碍治疗的潜力.

关键词:
在 AAV44.9 中使用.这是PCCA的PCCA.心脏热带性心脏热带性基因组编辑 基因组编辑鼠标模型 鼠标模型鼠标模型新生儿基因治疗新生儿基因治疗有机酸性疾病有机酸性疾病酸血症 酸血症尼尔可阿碳酸酶缺乏症系统性基因治疗.

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